Global epigenetic regulation and capsid delivery license agreement with Genentech
Click here to view
Careers
Core Values
Diversity, Equity and Inclusion
Open Positions
Employee Benefits
Clinical Trials
Contact
Science
Technology Platforms
Zinc Finger Platform
Delivery Platform
Therapeutic Applications
Neurology
Oncology
Presentations
Publications
Programs
Pipeline
Clinical Programs
Hemophilia A
Fabry Disease
Renal transplantation
Sickle Cell Disease
Clinical Trials
Collaborations & Partners
Patients
Our Commitment
Expanded Access Policy
Bioethics
Patient Stories
Bryan – Fabry Disease
Lori – Fabry Disease
Matt – Fabry Disease
Elizabeth – Fabry Disease
Israel – Hemophilia A
Miles – Hemophilia A
Trevor – Prion Disease
Sharron – Renal Transplant
Patient Focus
Resources
Education
Organizations
Investors & Media
Overview
News Releases
Events
Presentations
ESG
About
Overview
Mission & Vision
Executive Leadership
Board of Directors
Latest News
News Releases
Events
Presentations
Contact
Science
Technology Platforms
Zinc Finger Platform
Delivery Platform
Therapeutic Applications
Neurology
Oncology
Presentations
Publications
Programs
Pipeline
Clinical Programs
Hemophilia A
Fabry Disease
Renal transplantation
Sickle Cell Disease
Clinical Trials
Collaborations & Partners
Patients
Our Commitment
Expanded Access Policy
Bioethics
Patient Stories
Bryan – Fabry Disease
Lori – Fabry Disease
Matt – Fabry Disease
Elizabeth – Fabry Disease
Israel – Hemophilia A
Miles – Hemophilia A
Trevor – Prion Disease
Sharron – Renal Transplant
Patient Focus
Resources
Education
Organizations
Investors & Media
Overview
News Releases
Events
Presentations
ESG
About
Overview
Mission & Vision
Executive Leadership
Board of Directors
Latest News
News Releases
Events
Presentations
Contact
LinkedIn
Twitter
Hemophilia B
First-in-human in vivo genome editing via AAV-zinc finger nucleases for mucopolysaccharidosis I/II and
hemophilia B
November 1st, 2022